Our goals are to expand access to genomic testing and develop cutting-edge genomic testing to help more families get answers ...

Patient Advocacy Initiative staff speak on Notre Dame’s Rare Disease Day event and impact for the rare disease community.

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, ...

Rare eye diseases are the leading cause of untreatable blindness in Europe and affect people of all ages. The RESTORE VISION ...

ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...

For the first time, Hospital Kuala Lumpur (HKL) is proudly hosting the annual Rare Disease Day 2025 celebration to raise ...

And today we have two segments on rare diseases. Later, we'll hear from NFL legend, Boomer Esiason. But first, I want to welcome Dr. Marshall Summar, CEO of Uncommon Cures, and Pamela Gavin, CEO of ...

Zevra used the awareness day to roll out the “Learn NPC, Read Between the Signs” disease awareness campaign. The biotech won ...

Rare Disease Day, observed on February 28th, aims to raise awareness about rare diseases and the challenges faced by patients ...

Williams, who has performed the National Anthem at baseball stadiums as part of Boehringer Ingelheim’s Breathless campaign, ...

Donna Reynolds of Richmond knows all too well the devastating toll that a rare disease can take, not just on an individual, ...

Fanconi anemia (FA) is a rare genetic disorder impairing bone marrow's blood cell production, leading to severe health issues ...